Meeting Program

Title: Molecular therapies for liver disease in alpha-1 antitrypsin deficiency

Period: September 20-21, 2019


Nicola Brunetti-Pierri
Telethon Institute of Genetics and Medicine, IT

Pasquale Piccolo
Telethon Institute of Genetics and Medicine, IT

Pavel Strnad
RWTH Aachen, DE


Nedim Hadzic
King's College London, UK

Ron Kopito
Stanford University, US

David Lomas
University College London, UK

Christian Mueller
University of Massachusetts, US

Jeffrey Teckman
Saint Louis University, US


September 20th, 2109

10.30 Welcome and TIGEM presentation

11.00 Opening Lecture: – Title to be defined - Jeffrey Teckman

Session 1: Clinical spectrum of liver disease in AATD

12:00 "Clinical spectrum of AATD in childhood" - Nedim Hadzic
12:40 ‘’Liver phenotype of adults with AATD”- Pavel Strnad
13:20 Selected talk: “Fibroscan cap in AATD: analysis in an unselected consecutive population” - Federica Benini

13:35 Lunch

Session 2: Therapeutic approaches to liver disease in AATD

14:40 Title to be defined - David Lomas

15:20 Coffee break

15:50 Title to be defined - Proteostasis
16:15 Title to be defined - Arrowhead

20:00 Social dinner

September 21st, 2109

08:30 Keynote lecture: “Functional genomic dissection of protein homeostasis networks in the endoplasmic reticulum” - Ron Kopito

Session 3: New therapeutic targets: recent advances from basic science

9:30 “Non apoptotic cell death mechanisms in the liver pathophysiology of AATD” – Motti Gerlic
10:00 “JNK and CHOP activation aggravates liver disease due to mutant Z alpha-1 antitrypsin’’ - Nicola Brunetti-Pierri

10:30 Coffee break

11:00 “MicroRNA profiling unravels upregulation of miR-34c driven by JNK and FOXO3 in livers expressing mutant Z α1-antitrypsin” - Pasquale Piccolo
11:30 Selected talk: - “Role of p62 in the pathogenesis of liver disease due to Z alpha-1 antitrypsin Z alpha-1 antitrypsin” - Sergio Attanasio
11:45 Selected talk: ‘’Liver fibrosis and cirrhosis in PiZ mice’’- Lisa Bewersdorf
12:00 Selected talk: “Multiple decision points determine the cellular fate of the major pathogenic α1-antitrypsin variant” – Riccardo Ronzoni

12:15 Light lunch

Session 4: New tools for therapy discovery and development

13:00 Title to be defined - Christian Mueller
13:30 Selected talk: “Organoids as a model for AATD liver disease” – Beatriz Martinez-Delgado
13:45 Selected talk: “Structural analysis of alpha-1 antitrypsin polymers extracted from an explanted MZ heterozygote liver” – Mattia Laffranchi
14:00 Selected talk: “Developing function-blocking monoclonal antibodies against alpha1-antitrypsin to understand its immunomodulatory functions” – Emanuela d’Acunto

14:15 Coffee break

Session 4: Patients session

14:45: ’’Looking for 120000 Alphas in Europe – the situation of patients and patient associations today” - Frank Willersinn and members of patients’ organizations

15:30 Conclusion and farewell

Application period

May 1 – July 15, 2019

Abstract submission period

April 1 – June 30, 2019

Participation fees:

Academics/Principal Investigators: 100€
Student/Post-docs: 50€